A Phase I/II Study of Nula-cel in Autologous CD34+ Hematopoietic Stem Cells to Convert HbS to HbA for Treating Severe Sickle Cell Disease
Kamau Therapeutics
Summary
This study is a first-in-human, single-arm, open-label Phase I/II study of nula-cel in approximately 15 participants, diagnosed with severe Sickle Cell Disease. The primary objective is to evaluate safety of the treatment in this patient population, as well as preliminary efficacy and pharmacodynamic data.
Description
Participants diagnosed with severe SCD will receive nula-cel via IV infusion following myeloablative conditioning in an autologous HSCT setting.
Eligibility
- Age range
- 12–40 years
- Sex
- All
- Healthy volunteers
- No
Inclusion Criteria: * ≥12 to ≤ 40 years * Severe disease, as defined by having experienced at least one of the following SCD-related events despite appropriate supportive care measures: * recurrent severe VOC (≥ 4 episodes in the preceding 2 years) * ACS (≥ 2 episodes in the prior 2 years with at least one episode in the past year) * Lansky/Karnofsky performance status of ≥ 80 Exclusion Criteria: * Available 10/10 HLA-matched sibling donor * Prior HSCT or gene therapy * Prior or current malignancy or myeloproliferative or a significant coagulation or immunodeficiency disorder * Clinically s…
Interventions
- Geneticnula-cel Drug Product
nula-cel is administered via IV infusion following a myeloablative conditioning regimen
Locations (4)
- Children's Hospital Los AngelesLos Angeles, California
- Lucile Packard Children's HospitalPalo Alto, California
- Washington UniversitySt Louis, Missouri
- Nationwide Children's HospitalColumbus, Ohio